Long term follow-up for patients with inborn errors of immunity treated with autologous ex vivo gene modified CD34+ advanced therapies at Great Ormond Street Hospital
Medical condition: X-linked SCID (SCID-X1); p47 deficient Autosomal Recessive Chronic Granulomatous Disease (AR-CGD) and Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)
Patients will continue to receive routine medical care and follow-up appointments at GOSH, just as they normally would. This study will involve reviewing medical records and collecting information from standard medical check-ups. In some cases, additional blood samples may be taken. If needed, a bone marrow sample may be collected, but this would only happen in rare cases and would be discussed in advance with the patient and their family.
1. Participants must have previously received a CD34+ ATIMP through a clinical trial or compassionate use program at GOSH. 2. The patient displays persistent detectable gene marking. 3. The patient is not followed-up on another LTFU study for the same condition. 4. The patient or their guardian can provide informed consent. 5. Inclusion can be prospective or retrospective.
Refusal to sign informed consent